MX2020013293A - Guías modificadas de arn para edición génica. - Google Patents
Guías modificadas de arn para edición génica.Info
- Publication number
- MX2020013293A MX2020013293A MX2020013293A MX2020013293A MX2020013293A MX 2020013293 A MX2020013293 A MX 2020013293A MX 2020013293 A MX2020013293 A MX 2020013293A MX 2020013293 A MX2020013293 A MX 2020013293A MX 2020013293 A MX2020013293 A MX 2020013293A
- Authority
- MX
- Mexico
- Prior art keywords
- gene editing
- guidelines
- modified rna
- vitro
- vivo
- Prior art date
Links
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Medicinal Chemistry (AREA)
- Crystallography & Structural Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Mycology (AREA)
- Public Health (AREA)
- Virology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862682838P | 2018-06-08 | 2018-06-08 | |
| US201862682820P | 2018-06-08 | 2018-06-08 | |
| PCT/US2019/036160 WO2019237069A1 (en) | 2018-06-08 | 2019-06-07 | Modified guide rnas for gene editing |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2020013293A true MX2020013293A (es) | 2021-05-12 |
Family
ID=68770688
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2020013293A MX2020013293A (es) | 2018-06-08 | 2019-06-07 | Guías modificadas de arn para edición génica. |
| MX2025001112A MX2025001112A (es) | 2018-06-08 | 2020-12-07 | Guias modificadas de arn para edicion genica |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2025001112A MX2025001112A (es) | 2018-06-08 | 2020-12-07 | Guias modificadas de arn para edicion genica |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US20210087568A1 (enExample) |
| EP (1) | EP3802828A4 (enExample) |
| JP (2) | JP7832761B2 (enExample) |
| KR (2) | KR20260030838A (enExample) |
| CN (1) | CN112567036A (enExample) |
| AU (2) | AU2019282824C1 (enExample) |
| BR (1) | BR112020024731A2 (enExample) |
| CA (1) | CA3102950A1 (enExample) |
| CO (1) | CO2021000051A2 (enExample) |
| IL (2) | IL278822B1 (enExample) |
| MX (2) | MX2020013293A (enExample) |
| PH (1) | PH12020552100A1 (enExample) |
| SA (1) | SA520420729B1 (enExample) |
| SG (1) | SG11202011539VA (enExample) |
| TW (1) | TW202016306A (enExample) |
| WO (1) | WO2019237069A1 (enExample) |
Families Citing this family (60)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RU2019126483A (ru) | 2017-01-23 | 2021-02-24 | Ридженерон Фармасьютикалз, Инк. | Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение |
| KR20190139869A (ko) | 2017-04-11 | 2019-12-18 | 리제너론 파마슈티칼스 인코포레이티드 | 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정 |
| US11866726B2 (en) | 2017-07-14 | 2024-01-09 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
| CN110891420B (zh) | 2017-07-31 | 2022-06-03 | 瑞泽恩制药公司 | Cas转基因小鼠胚胎干细胞和小鼠及其应用 |
| KR20200033259A (ko) | 2017-07-31 | 2020-03-27 | 리제너론 파마슈티칼스 인코포레이티드 | 생체내에서 CRISPR/Cas-매개된 파괴 또는 삭제 및 외인성 도너 핵산과의 CRISPR/Cas-유도된 재조합을 평가하기 위한 방법 및 조성물 |
| US20190098879A1 (en) | 2017-09-29 | 2019-04-04 | Regeneron Pharmaceuticals, Inc. | Non-Human Animals Comprising A Humanized TTR Locus And Methods Of Use |
| AU2018348195B2 (en) | 2017-10-11 | 2025-05-15 | Regeneron Pharmaceuticals, Inc. | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
| EP3592140A1 (en) | 2018-03-19 | 2020-01-15 | Regeneron Pharmaceuticals, Inc. | Transcription modulation in animals using crispr/cas systems |
| AU2019239971B2 (en) | 2018-03-21 | 2025-09-11 | Regeneron Pharmaceuticals, Inc. | 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof |
| EP3823633A4 (en) | 2018-06-29 | 2023-05-03 | Editas Medicine, Inc. | SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO |
| AU2019360270B2 (en) | 2018-10-18 | 2025-08-07 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor IX. |
| JP7631215B2 (ja) * | 2019-03-28 | 2025-02-18 | インテリア セラピューティクス，インコーポレイテッド | Ｔｔｒガイドｒｎａと、ｒｎａガイドｄｎａ結合剤をコードするポリヌクレオチドとを含む組成物および方法 |
| US11891618B2 (en) | 2019-06-04 | 2024-02-06 | Regeneron Pharmaceuticals, Inc. | Mouse comprising a humanized TTR locus with a beta-slip mutation and methods of use |
| MX2021015122A (es) | 2019-06-07 | 2022-04-06 | Regeneron Pharma | Animales no humanos que comprenden un locus de albumina humanizado. |
| CN113906134B (zh) | 2019-06-14 | 2025-06-24 | 瑞泽恩制药公司 | Tau蛋白病模型 |
| WO2021026318A2 (en) * | 2019-08-06 | 2021-02-11 | The Penn State Research Foundation | Engineered crispr/cas9 systems for simultaneous long-term regulation of multiple targets |
| US12521451B2 (en) | 2019-11-08 | 2026-01-13 | Regeneron Pharmaceuticals, Inc. | CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy |
| EP4438729A3 (en) * | 2019-12-09 | 2025-03-26 | Caribou Biosciences, Inc. | Crispr abasic restricted nucleotides and crispr accuracy via analogs |
| EP4073249A1 (en) * | 2019-12-11 | 2022-10-19 | Intellia Therapeutics, Inc. | Modified guide rnas for gene editing |
| BR112022015380A2 (pt) | 2020-02-07 | 2022-09-27 | Intellia Therapeutics Inc | Composições e métodos para edição de gene de calicreína (klkb1) |
| MX2022012683A (es) * | 2020-04-09 | 2023-01-11 | Verve Therapeutics Inc | Edicion de bases de pcsk9 y metodos de uso de la misma para el tratamiento de enfermedades. |
| CN115956120A (zh) * | 2020-05-12 | 2023-04-11 | 马萨诸塞大学 | 用于crispr基因组编辑的修饰的指导rna |
| MX2023006878A (es) | 2020-12-11 | 2023-07-31 | Intellia Therapeutics Inc | Composiciones y métodos para reducir el mhc de clase ii en una célula. |
| IL303506A (en) | 2020-12-11 | 2023-08-01 | Intellia Therapeutics Inc | Polynucleotides, compositions, and methods for genome editing involving deamination |
| WO2022140587A1 (en) | 2020-12-23 | 2022-06-30 | Intellia Therapeutics, Inc. | Compositions and methods for genetically modifying ciita in a cell |
| CR20230320A (es) | 2020-12-23 | 2023-10-23 | Intellia Therapeutics Inc | Composiciones y métodos para reducir los hla-a en una célula |
| EP4288089A2 (en) | 2021-02-08 | 2023-12-13 | Intellia Therapeutics, Inc. | T-cell immunoglobulin and mucin domain 3 (tim3) compositions and methods for immunotherapy |
| EP4288525A1 (en) | 2021-02-08 | 2023-12-13 | Intellia Therapeutics, Inc. | Natural killer cell receptor 2b4 compositions and methods for immunotherapy |
| CN117042793A (zh) | 2021-02-08 | 2023-11-10 | 因特利亚治疗公司 | 用于免疫疗法的淋巴细胞活化基因3(lag3)组合物和方法 |
| AU2022227650A1 (en) * | 2021-02-25 | 2023-10-12 | Celyntra Therapeutics Sa | Compositions and methods for targeting, editing, or modifying genes |
| JP2024534114A (ja) | 2021-08-24 | 2024-09-18 | インテリア セラピューティクス，インコーポレイテッド | 細胞療法用のプログラム細胞死タンパク質１（ｐｄ１）組成物及び方法 |
| JP2024540723A (ja) | 2021-11-03 | 2024-11-01 | インテリア セラピューティクス，インコーポレーテッド | 免疫療法のためのｃｄ３８組成物及び方法 |
| IL312507A (en) * | 2021-11-03 | 2024-07-01 | Intellia Therapeutics Inc | Modified guide rnas for gene editing |
| KR20240117571A (ko) | 2021-12-08 | 2024-08-01 | 리제너론 파마슈티칼스 인코포레이티드 | 돌연변이 마이오실린 질환 모델 및 이의 용도 |
| WO2023185697A2 (en) * | 2022-03-29 | 2023-10-05 | Accuredit Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treatment of transthyretin amyloidosis |
| CN114574598B (zh) * | 2022-04-02 | 2023-08-08 | 中国农业科学院北京畜牧兽医研究所 | 鉴定或辅助鉴定猪5/6肋眼肌面积的方法 |
| KR20250037788A (ko) * | 2022-06-15 | 2025-03-18 | 빔 테라퓨틱스, 인크. | 보체 활성화를 감소시키기 위한 조성물 및 방법 |
| CN119384498A (zh) | 2022-06-16 | 2025-01-28 | 因特利亚治疗公司 | 用于对细胞进行遗传修饰的方法和组合物 |
| AU2023293131A1 (en) | 2022-06-16 | 2024-12-12 | Intellia Therapeutics, Inc. | Compositions and methods for reducing mhc class i in a cell |
| WO2024006955A1 (en) | 2022-06-29 | 2024-01-04 | Intellia Therapeutics, Inc. | Engineered t cells |
| WO2024003810A1 (en) * | 2022-06-30 | 2024-01-04 | Geneditbio Limited | Guide rna with chemical modifications |
| KR20250124819A (ko) | 2022-12-21 | 2025-08-20 | 인텔리아 테라퓨틱스, 인크. | 프로단백질 전환효소 서브틸리신 켁신 9(pcsk9) 편집을 위한 조성물 및 방법 |
| CN120418427A (zh) | 2022-12-23 | 2025-08-01 | 因特利亚治疗公司 | 用于基因组编辑的系统和方法 |
| TW202436622A (zh) | 2023-03-06 | 2024-09-16 | 美商英特利亞醫療公司 | 用於ｂ型肝炎病毒（ｈｂｖ）基因體編輯之組合物及方法 |
| WO2024186971A1 (en) | 2023-03-07 | 2024-09-12 | Intellia Therapeutics, Inc. | Cish compositions and methods for immunotherapy |
| AU2024270764A1 (en) | 2023-05-15 | 2025-12-04 | Nchroma Bio, Inc. | Compositions and methods for epigenetic regulation of hbv gene expression |
| CN119265190A (zh) * | 2023-07-06 | 2025-01-07 | 上海交通大学 | 一种靶向TTR的saCas9 sgRNA及其修饰方式 |
| WO2025038646A1 (en) | 2023-08-14 | 2025-02-20 | Intellia Therapeutics, Inc. | Cd70 car-t compositions and methods for cell-based therapy |
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| KR102888521B1 (ko) * | 2015-04-06 | 2025-11-19 | 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 | Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna |
| US11279928B2 (en) * | 2015-06-29 | 2022-03-22 | Massachusetts Institute Of Technology | Compositions comprising nucleic acids and methods of using the same |
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| WO2017136794A1 (en) * | 2016-02-03 | 2017-08-10 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide rna and its applications |
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| WO2019067910A1 (en) * | 2017-09-29 | 2019-04-04 | Intellia Therapeutics, Inc. | POLYNUCLEOTIDES, COMPOSITIONS AND METHODS FOR GENOMIC EDITION |
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